Ribozymes in medicine
Languages of publication
Catalytic nucleic acids, ribozymes and deoxyribozymes can act as efficient ribonucleases and degrade target RNA molecules by complementary Watson-Crick base pairing and catalytic cleavage of their phosphodiester internucleotide bonds. This ability makes ribozymes and deoxyribozymes potent molecular tools for therapeutic applications. Recent achievements in ribozymes design and technology enable the preparation of ribozymes which can be efficiently expressed in cellular systems, co-localize with the target mRNA and exhibit high intracellular activity. Several examples of preclinical and clinical trials of ribozymes directed toward viral genes (HBV, HCV, HIV-1) and oncogenes are discussed in this review.
Publication order reference
S. Antoszczyk, Zaklad Chemii Bioorganicznej, Centrum Badan Molekularnych i Makromolekularnych, Polska Akademia Nauk, ul. Sienkiewicza 112, 90-363 Lodz, Poland